CN110418800A - 治疗溶酶体紊乱的方法 - Google Patents

治疗溶酶体紊乱的方法 Download PDF

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Publication number
CN110418800A
CN110418800A CN201880018325.9A CN201880018325A CN110418800A CN 110418800 A CN110418800 A CN 110418800A CN 201880018325 A CN201880018325 A CN 201880018325A CN 110418800 A CN110418800 A CN 110418800A
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China
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leu
transmembrane protein
ser
val
lysosomal transmembrane
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Chinese (zh)
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斯蒂芬妮·切尔奎
埃里克·阿德勒
塞尔维亚·埃文斯
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University of California
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University of California
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Environmental Sciences (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
CN201880018325.9A 2017-03-15 2018-03-15 治疗溶酶体紊乱的方法 Pending CN110418800A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762471741P 2017-03-15 2017-03-15
US62/471,741 2017-03-15
US201762507713P 2017-05-17 2017-05-17
US62/507,713 2017-05-17
PCT/US2018/022598 WO2018170239A1 (en) 2017-03-15 2018-03-15 Methods of treating lysosomal disorders

Publications (1)

Publication Number Publication Date
CN110418800A true CN110418800A (zh) 2019-11-05

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CN201880018325.9A Pending CN110418800A (zh) 2017-03-15 2018-03-15 治疗溶酶体紊乱的方法

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US (2) US11806367B2 (https=)
EP (1) EP3596111A4 (https=)
JP (3) JP7233375B2 (https=)
CN (1) CN110418800A (https=)
AU (2) AU2018236353B2 (https=)
CA (1) CA3056617A1 (https=)
IL (1) IL269082A (https=)
WO (1) WO2018170239A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109880849A (zh) * 2019-01-03 2019-06-14 四川大学 靶向肿瘤相关巨噬细胞的GHOST-shRNA表达载体复合物及其应用
CN114934066A (zh) * 2022-04-28 2022-08-23 深圳临研医学有限公司 石骨症的基因编辑体系及其应用
CN115461459A (zh) * 2020-07-08 2022-12-09 纽洛可遗传学有限公司 新型内含子片段
CN118388623A (zh) * 2024-06-28 2024-07-26 首都医科大学 Sialin蛋白在调控细胞线粒体功能中的应用

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3056617A1 (en) 2017-03-15 2018-09-20 The Regents Of The University Of California Methods of treating lysosomal disorders
KR102918544B1 (ko) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
WO2020160458A1 (en) 2019-02-01 2020-08-06 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
WO2020179931A1 (ja) * 2019-03-07 2020-09-10 国立大学法人 東京医科歯科大学 ハイスループット遺伝子編集技術
US12319928B2 (en) * 2019-03-21 2025-06-03 The Regents Of The University Of Colorado, A Body Corporate Methods of treating Danon disease
WO2020210218A1 (en) * 2019-04-12 2020-10-15 The Regents Of The University Of California Methods for treating inherited eye defects
WO2021073607A1 (en) * 2019-10-17 2021-04-22 Westlake Therapeutics (Hangzhou) Co. Limited Engineering red blood cells for treatment of lysosomal storage diseases
GB202003536D0 (en) * 2020-03-11 2020-04-29 Ucl Business Ltd Gene therapy

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014011237A1 (en) * 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
CN106459995A (zh) * 2013-11-07 2017-02-22 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
HK1208371A1 (en) 2012-10-09 2016-03-04 桑比欧公司 Methods and compositions for treatment of retinal degeneration
US10155794B2 (en) 2013-07-16 2018-12-18 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to CEP290
PE20180772A1 (es) * 2015-07-01 2018-05-07 Univ California Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa
US10670862B2 (en) 2015-07-02 2020-06-02 Microsoft Technology Licensing, Llc Diffractive optical elements with asymmetric profiles
JP7089282B2 (ja) * 2015-07-20 2022-06-22 アンジオクライン・バイオサイエンス・インコーポレイテッド 幹細胞移植のための方法および組成物
CA3056617A1 (en) 2017-03-15 2018-09-20 The Regents Of The University Of California Methods of treating lysosomal disorders

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014011237A1 (en) * 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
CN106459995A (zh) * 2013-11-07 2017-02-22 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
HARRISON F等: ""Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis", 《MOL THER.》, pages 433 - 444 *
HARRISON F等: "Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis", 《MOL THER.》 *
HARRISON F等: "Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis", 《MOL THER.》, vol. 21, no. 2, 23 October 2012 (2012-10-23), pages 433 - 444, XP055542991, DOI: 10.1038/mt.2012.214 *
朱碧溱等: "以低分子质量蛋白尿为主要表现的遗传性肾小管疾病", 《实用儿科临床杂志》 *
朱碧溱等: "以低分子质量蛋白尿为主要表现的遗传性肾小管疾病", 《实用儿科临床杂志》, no. 11, 5 June 2011 (2011-06-05), pages 81 - 82 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109880849A (zh) * 2019-01-03 2019-06-14 四川大学 靶向肿瘤相关巨噬细胞的GHOST-shRNA表达载体复合物及其应用
CN115461459A (zh) * 2020-07-08 2022-12-09 纽洛可遗传学有限公司 新型内含子片段
CN114934066A (zh) * 2022-04-28 2022-08-23 深圳临研医学有限公司 石骨症的基因编辑体系及其应用
CN118388623A (zh) * 2024-06-28 2024-07-26 首都医科大学 Sialin蛋白在调控细胞线粒体功能中的应用
CN118388623B (zh) * 2024-06-28 2024-10-08 首都医科大学 Sialin蛋白在调控细胞线粒体功能中的应用

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AU2018236353B2 (en) 2023-12-21
JP2020511470A (ja) 2020-04-16
EP3596111A1 (en) 2020-01-22
IL269082A (en) 2019-11-28
US20240009247A1 (en) 2024-01-11
EP3596111A4 (en) 2021-01-06
AU2024201797A1 (en) 2024-04-11
CA3056617A1 (en) 2018-09-20
AU2018236353A1 (en) 2019-09-26
JP2023052475A (ja) 2023-04-11
US11806367B2 (en) 2023-11-07
WO2018170239A1 (en) 2018-09-20
US20210161966A1 (en) 2021-06-03
JP7233375B2 (ja) 2023-03-06
JP2025039642A (ja) 2025-03-21

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